BR112021019880A2 - Gene therapies for lysosomal disorders - Google Patents

Gene therapies for lysosomal disorders

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Publication number
BR112021019880A2
BR112021019880A2 BR112021019880A BR112021019880A BR112021019880A2 BR 112021019880 A2 BR112021019880 A2 BR 112021019880A2 BR 112021019880 A BR112021019880 A BR 112021019880A BR 112021019880 A BR112021019880 A BR 112021019880A BR 112021019880 A2 BR112021019880 A2 BR 112021019880A2
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BR
Brazil
Prior art keywords
disease
gene therapies
parkinson
lysosomal disorders
gaucher
Prior art date
Application number
BR112021019880A
Other languages
Portuguese (pt)
Inventor
Asa Abeliovich
Franz Hefti
Herve Rhinn
Laura Heckman
Original Assignee
Prevail Therapeutics Inc
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Publication date
Application filed by Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc
Publication of BR112021019880A2 publication Critical patent/BR112021019880A2/en

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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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Abstract

terapias gênicas para distúrbios lisossomais. a presente invenção refere-se a composições e métodos para o tratamento de doenças associadas à função lisossomal aberrante, como o mal de parkinson e a doença de gaucher. a presente invenção refere-se a construtos de expressão que compreendem um transgene que codifica beta-glicocerebrosidase, um rna inibidor que direciona a alfa-sinucleína ou uma combinação dos anteriores. a divulgação fornece adicionalmente métodos de tratamento da doença de gaucher, mal de parkinson ou outras sinucleinopatias pela administração de tais construtos de expressão a um indivíduo em necessidade do mesmo.gene therapies for lysosomal disorders. The present invention relates to compositions and methods for treating diseases associated with aberrant lysosomal function, such as parkinson's disease and gaucher's disease. the present invention relates to expression constructs comprising a transgene encoding beta-glucocerebrosidase, an inhibitor RNA that targets alpha-synuclein, or a combination of the foregoing. the disclosure further provides methods of treating gaucher's disease, parkinson's disease or other synucleinopathies by administering such expression constructs to a subject in need thereof.

BR112021019880A 2019-04-10 2020-04-10 Gene therapies for lysosomal disorders BR112021019880A2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962831840P 2019-04-10 2019-04-10
US202062990246P 2020-03-16 2020-03-16
PCT/US2020/027788 WO2020210713A1 (en) 2019-04-10 2020-04-10 Gene therapies for lysosomal disorders

Publications (1)

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BR112021019880A2 true BR112021019880A2 (en) 2022-02-15

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US (2) US11903985B2 (en)
EP (1) EP3953378A1 (en)
JP (1) JP2022526021A (en)
KR (1) KR20220006527A (en)
CN (1) CN114026115A (en)
AU (1) AU2020270984A1 (en)
BR (1) BR112021019880A2 (en)
CA (1) CA3136117A1 (en)
IL (1) IL286901A (en)
MX (1) MX2021012184A (en)
WO (1) WO2020210713A1 (en)

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